Trial of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease or Primary Tauopathies

A vizsgálat adatai

Ki végzi

Arrowhead Pharmaceuticals

Azonosító

NCT07221344

A regiszteradat frissítve

2026. november 10.

Eredeti leírás

Trial to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ARO-MAPT-SC compared to placebo in adult healthy volunteers and in participants with early Alzheimer's disease (AD), defined as mild cognitive impairment due to AD and mild AD dementia, as well as primary tauopathies, including microtubule associated protein tau -related frontotemporal dementia (MAPT-FTD) and progressive supranuclear palsy- Richardson syndrome (PSP-RS).A teljes eredeti leírásKevesebb

A Phase 1/2a Placebo-controlled Dose-escalating Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-MAPT-SC in Healthy Participants and Participants With Early Alzheimer's Disease or Primary Tauopathies

Trial to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ARO-MAPT-SC compared to placebo in adult healthy volunteers and in participants with early Alzheimer's disease (AD), defined as mild cognitive impairment due to AD and mild AD dementia, as well as primary tauopathies, including microtubule associated protein tau -related frontotemporal dementia (MAPT-FTD) and progressive supranuclear palsy- Richardson syndrome (PSP-RS).

Bevonási feltételek: Body mass index between 18.0 and 35.0 kilograms (kg)/square meter (m\^2) at Screening; Not pregnant or breast-feeding; Able and willing to provide written informed consent prior to the performance of any trial specific procedures; Participants of childbearing potential must agree to use highly effective contraception in addition to a condom during the trial and for at least 90 days following the end of the trial or last dose of trial drug, whichever is later; participants must not donate sperm or eggs during the trial and for at least 90 days following the end of the trial or last dose of trial drug whichever is later; Adults aged 50 to ≤80 years of age with a clinical diagnosis of early AD and plasma, CSF, or imaging biomarkers consistent with the diagnosis; If participant is on non-disease-modifying AD medications, the doses must be stable for ≥8 weeks prior to Screening; Participants with early AD are not required to be on AD medications; Participants previously treated or currently receiving anti-amyloid therapies, including lecanemab and donanemab, are not eligible; Adults ≥18 years of age with Clinical Dementia Rating plus National Alzheimer's Coordinating Center Frontotemporal Lobar Degeneration-Neuropsychiatric and Motor domains Sum of Boxes Global Score of 0.5 or 1 at Screening; Frontotemporal Lobar Degeneration (F-causative MAPT mutation, based on source verifiable medical records; If the participant is on medications for symptomatic management of MAPT-FTD, the doses must be stable for ≥8 weeks prior to Screening; Adults ≥41 to ≤86 years of age with clinical diagnosis of probable PSP-RS phenotype as defined by the 2017 Movement Disorder Society criteria (Höglinger 2017) with symptom onset ≥40 to ≤85 years of age; Presence of PSP symptoms ≤3 years at Screening (occurring newly and subsequently progressing during the clinical course in the absence of another identifiable cause) as determined by the Investigator; Mini-Mental Status Examination (MMSE) score of 20 to 30 (inclusive) at Screening; Able to walk at least 10 steps with minimal assistance (eg, one arm for safety, but not postural support); Reside at home or in the community (assisted living is acceptable); outside of a skilled nursing facility or dementia care facility at the time of screening, with no planned admissions to such a facility at time of Screening; If the participant is on medications for symptomatic management of PSP-RS, the doses must be stable for ≥8 weeks prior to Screening; Have a reliable and competent caregiver or trial partner who is ≥18 years of age, able and willing to accompany the participant to trial visits involving informant-based assessments, to be available to site staff by telephone as needed, and in the opinion of the Investigator, be sufficiently familiar with the participant throughout the trial in order to provide accurate and reliable information relevant to trial outcome measures.

Kizáró feltételek: Human immunodeficiency virus (HIV) infection (seropositive at Screening); Seropositive for hepatitis B (HBV) or hepatitis C (HCV) at Screening; Clinically significant cardiac, liver, or renal disease; Known allergy or possible allergy to either ARO-MAPT-SC or to its excipients; Note: Additional inclusion/exclusion criteria may apply per protocol; Blood pressure outside of specified range in the protocol; Intellectual disability or significant behavioral neuropsychiatric manifestation; Any contraindications to lumbar puncture.

ClinicalTrials.gov NCT07221344